Orphalan announces U.S. launch of drug to treat Wilson disease

liver rare disease Wilson

Orphalan SA, an international orphan drug development and commercialization company, has announced the commercial launch in the U.S. for Cuvrior. 

Cuvrior is a new trientine tetrahydrochloride (TETA-4HCl). It is now available for the treatment of adult patients with stable Wilson disease who are de-coppered and tolerant to D-penicillamine.

Cuvrior was approved by the United States Food and Drug Administration (FDA) and was granted Orphan Drug Exclusivity (ODE) in April 2022 for this patient group. 

Compared with the standard of care, Cuvrior facilitates precise dosing by providing a small, scored tablet in a blister pack. Additionally, its room temperature stability eliminates the need for refrigeration, making it a more practical solution for patients with busy lifestyles.

Orphalan drug available in Europe

Wilson disease is a rare inherited disorder of copper transport primarily affecting the liver and brain. Since 2019, Orphalan has commercialized its trientine tetrahydrochloride product in Europe under the name of Cuprior and approximately 1,000 Wilson disease patients have received treatment.

Orphalan’s Cuvrior was approved by the FDA on the basis of data from the company’s phase III CHELATE trial, the first prospective randomized trial comparing penicillamine with TETA-4HCl. During the trial, an assay was developed to measure non-caeruloplasmin bound copper (NCC), the free and potentially toxic pool of copper in the blood. Using this NCC measurement in patients previously receiving maintenance penicillamine therapy, TETA-4HCl was determined to be non-inferior to penicillamine at the primary endpoint of the study (24 weeks), with the same observation at the end of the extension phase of the study (one year from randomization).

Dmitry Paramonov, president of Orphalan US, said: “We are thrilled to introduce Cuvrior in the U.S., a safe and innovative treatment option for patients and caregivers managing Wilson disease. Our distribution partners have Cuvrior stocked and ready for immediate use. At Orphalan, our focus is on providing targeted solutions for rare diseases, as demonstrated by the successful launch of Cuprior in Europe. We are eager to expand availability of Cuvrior to as many patients as possible, underscoring our dedication to making a meaningful impact on the lives of rare disease patients worldwide.”

About Wilson disease

Wilson disease is a rare genetic disease that is characterized by gradual accumulation of dietary copper over time, possibly to life-threatening levels. Affected individuals are unable to effectively excrete copper naturally through the digestive tract. Common symptoms associated with Wilson disease include progressive liver dysfunction, neurological disorders such as severe tremors, and mental health deterioration. About 1 in 15 patients eventually need a liver transplant. Wilson disease affects nearly 1 in every 30,000 people worldwide.

Few companies are tackling the rare disease. Wilson Therapeutics, which was taken over by Alexion Pharmaceuticals in an $855 million deal in 2018, reported positive results from its WTX101 drug in 2016.

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